CERebrolysin In CADASIL

NCT05755997 | Active Not Recruiting Phase 2

• 2 other identifiers: EVER-CZ-04212022-002394-29
• Study Type: interventional
• Target: 30
• Locations: 1 country
• Sites: 1

Brief Summary

The objective of this trial is the global risk-benefit assessment of Cerebrolysin as compared to Placebo in patients with genetically proven CADASIL. In addition, a traditional approach will be taken based on an evaluation of the separate risk and benefit domains in comparison with placebo.

Conditions

Cadasil

Outcome Measures

Primary Outcomes (10)

• Change in cognitive battery (RAVLT)

  \- Rey Auditory Verbal Learning Test (AVLT)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery (ROCF: Copy, immediate recall)

  \- Rey-Osterrieth Complex Figure Test (ROCF): Copy, immediate recall

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery (Digit Symbol Coding, subscale of WAIS-PSI)

  \- Digit Symbol Coding (subscale of WAIS-PSI)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery (Digit Span: Digit backward (subscale of WAIS-WMI)

  \- Digit Span: Digit backward (subscale of WAIS-WMI)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery (Trail Making Test, Part B)

  \- Trail Making Test (Part B)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery (ROCF: Delayed recall)

  \- Rey-Osterrieth Complex Figure Test (ROCF): Delayed recall

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery (Stroop Color and Word Test - Prague Version)

  \- Stroop Color and Word Test - Prague Version (word/dots interference)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery (MoCA)

  \- Montreal Cognitive Assessment (MoCA)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in mood (Beck Depression Inventory-II)

  \- Beck Depression Inventory-II (BDI-II)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in imaging (White matter lesion volume)

  \- White matter lesion volume (MRI)

  Baseline, Month 12, Month 27

Secondary Outcomes (11)

• Change in cognitive battery, secondary outcome (Spatial Pattern Separation Task)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery, secondary outcome (Navigation Test Suite)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery, secondary outcome (Trail Making Test, Part A)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery, secondary outcome (Stroop Color and Word Test - Prague Version)

  Baseline, Month 6, Month 12, Month 21, Month 27

• Change in cognitive battery, secondary outcome (Symbol Search, subscale of WAIS-PSI)

  Baseline, Month 6, Month 12, Month 21, Month 27

Other Outcomes (28)

• Experimental (to be defined after study endpoint): Change in Serotonin level (hair, mean)

  Baseline, Month 12, Month 27

• Experimental (to be defined after study endpoint): Change in Oxytocin level (hair, mean)

  Baseline, Month 12, Month 27

• Experimental (to be defined after study endpoint): Change in Cortisol level (hair, mean)

  Baseline, Month 12, Month 27

Study Arms (2)

Study group 1

OTHER

Cerebrolysin - Placebo

Drug: Cerebrolysin; Drug: 0.9 % NaCl

Study group 2

OTHER

Placebo - Cerebrolysin

Drug: Cerebrolysin; Drug: 0.9 % NaCl

Interventions

Cerebrolysin DRUG

40 ml Cerebrolysin and 60 ml 0.9% NaCl per day for 4 days every month for 1 year

Also known as: Renacenz

Study group 1; Study group 2

0.9 % NaCl DRUG

100 ml 0.9% NaCl per day for 4 days every month for 1 year

Also known as: Sodium Chloride

Study group 1; Study group 2

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Patients of ≥18 years of age, all genders
• Diagnosis of CADASIL based on clinical symptoms, MRI, and genetic analysis
• MoCA \>11
• Adequate visual, auditory, and language skills (no language interpreter required) to follow study procedures
• Patient is not of childbearing potential (i.e. women are post-menopausal for two years, surgically sterile, or using adequate method of contraception)
• Patient participates voluntarily and gave written informed consent

You may not qualify if:

• Any significant neurological disease/conditions other than CADASIL
• Focal lesions that may be responsible for the cognitive status of the patient (e.g.
• infectious disease, space-occupying lesion, normal pressure hydrocephalus)
• Any other diseases/conditions that may affect compliance with the protocol, such as:
• severe psychiatric disorders within the last three months
• delusional symptoms
• history of schizophrenia, schizoaffective disorder, bipolar affective disorder
• major depressive disorder newly identified within eight weeks before screening
• history of alcohol or substance abuse or dependence within the past two years
• Any circumstances that -in the investigator's opinion- may result in the patient's non-compliance with study procedures, e.g. fragile or thin veins that prevent many i.v. infusions
• Any other disease/conditions that may affect the safety assessment, such as:
• history of systemic cancer within the past two years
• history of myocardial infarction in the past year or unstable or severe cardiovascular disease (including uncontrolled hypertension and/or history of unstable hypertension not compensated by antihypertensive therapy)
• any clinically significant laboratory abnormalities at screening
• uncontrolled insulin-requiring diabetes or non-insulin dependent diabetes mellitus (HbA1c \>87 mmol/mol)

Sponsors & Collaborators

• Ever Neuro Pharma GmbH: lead
• idv Datenanalyse & Versuchsplanung: collaborator
• XClinical GmbH: collaborator

Study Sites (1)

Motol University Hospital

Prague, 150 06, Czechia

Location

MeSH Terms

Conditions

CADASIL

Interventions

cerebrolysin; Sodium Chloride

Condition Hierarchy (Ancestors)

Cerebral Infarction; Brain Infarction; Brain Ischemia; Cerebrovascular Disorders; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Cerebral Small Vessel Diseases; Dementia, Vascular; Cerebral Arterial Diseases; Intracranial Arterial Diseases; Stroke; Dementia; Vascular Diseases; Cardiovascular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Infarction; Ischemia; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Necrosis

Intervention Hierarchy (Ancestors)

Chlorides; Hydrochloric Acid; Chlorine Compounds; Inorganic Chemicals; Sodium Compounds

Study Officials

• Aleš Tomek, MUDr., Ph.D.

  Motol University Hospital

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: CROSSOVER
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: A two-period cross-over design, in which participants will be randomly allocated to either the Verum-Control sequence (sequence 1) or the Control-Verum sequence (sequence 2) in a 1:1 randomization.

Study Record Dates

• First Submitted: February 14, 2023
• First Posted: March 6, 2023
• Study Start: November 29, 2023
• Primary Completion: March 31, 2026
• Study Completion (Estimated): December 31, 2026
• Last Updated: February 25, 2025

Record last verified: 2025-02

Data Sharing

• IPD Sharing: Will not share

Locations

CZ (1)